Liver-specific gene therapy based on self-complementary adeno-associated virus for lysosomal acid lipase deficiency

IntroductionLysosomal acid lipase deficiency is a rare, autosomal-recessive disorder caused by inactivating mutations of the lysosomal acid lipase gene and accumulation of cholesteryl esters and triglycerides in lysosomes. Treatment with recombinant lysosomal acid lipase is effective, but involves s...

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Hlavní autoři:	Ruolan Zhang, Jing Lou, Zheng Jin, Yanmei Ou, Ting He, Hezhi Qu, Yanqun Yang, Xiao Qu
Médium:	Artigo
Jazyk:	Inglês
Vydáno:	Frontiers Media S.A. 2026-07-01
Edice:	Frontiers in Pharmacology
Témata:	adeno-associated virus endoplasmic reticulum stress gene therapy lysosomal acid lipase deficiency mitochondrial dysfunction
On-line přístup:	https://www.frontiersin.org/articles/10.3389/fphar.2026.1822727/full
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